FDA Reboot Needed to Grow Innovation, Senate Panel Told

Former senator blasts FDA for failing to live up to 2012 reform mandate.

WASHINGTON -- A former senator told his ex-colleagues that, to grow investment and speed the progress of cures, the FDA must be more adaptive in its approval process, more transparent, and more collaborative.

Tom Coburn, MD, an Oklahoma Republican who retired from the Senate earlier this year, spoke at a Senate Committee on Commerce, Science and Transportation hearing on Tuesday. He and other witnesses discussed ways to increase investment in biomedical research, which committee leaders said has stagnated over the last few years.

Coburn has worked with or been affected by the FDA's behavior, beyond his experience as a legislator. As the former manager of an optical products company he dealt with the agency's approval process. As a physician, he watched patients suffer through long delays in seeing new drugs come to market, and as a patient himself, his interest in the FDA's behavior deepened.

"It becomes very personal. When you have advanced cancer ... new breakthroughs and new methods of approving new drugs are very important," Coburn said.

Coburn criticized the agency's slow progress. Pointing to the "last piece of major legislation," the Food and Drug Administration Safety and Innovation Act (FDASIA), he said, "I'd ask you to go look at what the FDA's done, what Congress has mandated and signed by the president 3 years ago, and what you will see is not much."

Coburn called the 21st Century Cures Act "a good start," but "[i]t's got to have some teeth in it" and should "force" the agency to collaborate with industry and to be more transparent.

While critical of the agency's slow-footedness, on the one hand, Coburn said Congress is partly to blame for "beating up" the agency in the past. For example, the painkiller Vioxx was pulled from the shelves because of a previously unrecognized harm -- it increased the risk of cardiovascular events.

"There is no way you can be perfect a 100% of the time," he said. "We've lost a great drug that had a rare side effect."

In the aftermath of something like the Vioxx incident, the FDA becomes even more risk-averse.

And this, said Coburn, is the crux of the problem. Everyone, from policymakers to regulators, is worried about what happens if something "goes wrong" but things are "already deeply wrong."

"Millions of Americans are suffering and dying from untreatable diseases or the lack of better treatment options. Not because it has to be that way but because we have a regulatory scheme that makes it that way."

Coburn called on the agency to reconsider its view of randomized control trials as "the gold standard" in the approval process. Instead he said the agency should look to biomarkers and mechanisms of action as evidence for approval.

Another witness at the hearing, Keith Yamamoto, MD, vice chancellor for research at the University of California San Francisco, seconded Coburn's remarks. He noted that investments in the sciences, particularly "precision medicine," can hasten discovery of biomarkers.

"Being able to massively collect data on genome sequences and correlate that with defects... is going to uncover the causes of rare diseases and allow us to attack them directly," he said.

In the past, drug companies have been slow to develop treatments for rare diseases because the rewards didn't seem to outweigh the effort. But if the mechanistic pathways of a disease have already been discovered, that would encourage drug companies to take the next steps.

Coburn also urged changes to intellectual property rules. He argued that the country is losing out because those who would finance new cures and devices aren't assured of recouping their investment because of the short life of intellectual property.

"Too many medicines or medical devices are never developed because it takes too long and costs too much to bring them to patients," Coburn said.

Lastly, he advocated for the FDA to allow informed patients to decide what level of risk they are comfortable with in testing new treatments.

Again, Yamamoto agreed, noting that the spectrum of acceptability would vary with the patient. "Cancer patients are much more willing to take risk on therapies than somebody with poison ivy."

After the hearing, committee chairman Sen. Ted Cruz (R-Tex.) told reporters, "I think and hope the members of this committee will continue to look for ways to work together to take the testimony we received today and to implement specific legislative steps."

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